FDA approves concizumab-mtci prophylaxis for hemophilia A and B
The indication is for patients 12 years and older hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors.
FDA approves concizumab-mtci prophylaxis for hemophilia A and B | Image Credit: © Calin - © Calin - stock.adobe.com.
The FDA has approved concizumab-mtci (Alhemo; Novo Nordisk) in patients aged 12 years and older with hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors, for routine prophylaxis to prevent or reduce the frequency of bleeding episodes.1
Concizumab-mtc is injected subcutaneously daily according to the FDA.1
Patients with the rare inherited bleeding disorders can bleed for longer periods of time after injury or surgery and could have spontaneous bleeding in muscles, joints, and organs, which can be life-threatening.1
In the United States, the FDA estimates that as many as 33,000 males are living with hemophilia, with hemophilia A being more common (80% to 85% of total hemophilia population) than hemophilia B.1
Concizumab-mtc is disigned to block the TFPI protein in the body that stops blood for clotting. By way of blocking TFPI, concizumab-mtc improves the production of thrombin—a protein that helps to clot the blood and prevent bleeding—when the other clotting factors are missing or deficient in the presence of inhibitors, according to Novo Nordisk.2
The development of inhibitors remains the most serious treatment-related complication for people living with hemophilia," said Amy Shapiro, MD, CEO and co-medical director at the Indiana Hemophilia & Thrombosis Center, Inc, in a statement.2
"For patients with inhibitors, especially in hemophilia B, their hemophilia may remain poorly controlled and pose a life-threatening risk," said Shapiro. "The approval of [concizumab-mtc]... provides a much-needed alternative to the current standard of care in hemophilia B with inhibitors, while offering patients with hemophilia A with inhibitors more treatment options, ultimately providing more patients with inhibitors the opportunity to personalize their care and address current treatment gaps."2
Safety and efficacy of concizumab-mtc were evaluated in a phase 3, open-label, multi-national, and multi-center trial (NCT04083781) that featured 91 adults and 42 adolescent male patients with hemophilia A or B. These patients had inhibitors who have been prescribed, or are in need of, treatment with therapies that bypass the inhibitor effect, according to the FDA.1
Efficacy was evaluated by comparison of the number of treated bleeding episodes between the concizumab-mtc treatment group and the no prophylaxis group.1
A ratio of the annualized bleeding rates (ABR) was estimated to 0.14, corresponding to a reduction in ABR of 86% (95% CI, 0.07 to 0.29, [P < 0.001]) for the [concizumab-mtc] treatment group compared to the no prophylaxis group, according to study results presented by Novo Nordisk.1,2
"As a supportive secondary efficacy endpoint, 64% of the patients randomized to receive [concizumab-mtc] prophylaxis treatment experienced zero treated spontaneous and traumatic bleeds during the first 24 weeks of treatment vs. 11% with no prophylaxis," stated Novo Nordisk.2
Injection site reactions and hives were the most common adverse reactions of concizumab-mtc, reported in at least 5% of patients.1
"The approval of [concizumab-mtc] signifies a remarkable achievement in prophylactic hemophilia treatment for individuals with inhibitors aged 12 years and older who, in some cases, currently have few options," said Anna Windle, SVP Clinical Development, Novo Nordisk, in the press release. "As the first treatment of its kind for this population, [concizumab-mtc] represents a significant step in helping to address the unmet needs of patients with hemophilia with inhibitors."2
References:
1. FDA approves drug to prevent or reduce the frequency of bleeding episodes for patients with hemophilia A with inhibitors or hemophilia B with inhibitors. FDA. Press release. December 20, 2024. Accessed December 23, 2024. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-prevent-or-reduce-frequency-bleeding-episodes-patients-hemophilia-inhibitors-or?utm_medium=email&utm_source=govdelivery
2. FDA approves Alhemo injection as once-daily prophylactic treatment to prevent or reduce the frequency of bleeding episodes for adults and children 12 years of age and older with hemophilia A or B with inhibitors. Novo Nordisk. Press release. December 20, 2024. Accessed December 23, 2024. https://www.prnewswire.com/news-releases/fda-approves-alhemo-injection-as-once-daily-prophylactic-treatment-to-prevent-or-reduce-the-frequency-of-bleeding-episodes-for-adults-and-children-12-years-of-age-and-older-with-hemophilia-a-or-b-with-inhibitors-302337790.html
